The European Commission has cleared Intercept's Ocaliva for the treatment of primary biliary cholangitis (PBC), giving patients the potential to access the first new therapy for the condition in nearly 20 years.
PBC is a rare, autoimmune cholestatic liver disease that primarily affecting women. If left untreated, survival of patients is significantly worse than the general population.
Ocaliva (obeticholic acid) is a highly selective agonist of the farnesoid X receptor (FXR), a nuclear receptor expressed in the liver and intestine that regulates bile acid, inflammatory, fibrotic and metabolic pathways.
In a Phase III clinical trial nearly half of patients (46 percent) in the titration group treated with Ocaliva in combination with UDCA achieved the primary endpoint of achieving a significant reduction in serum alkaline phosphatas (ALP) compared to 10 percent in the control group, while 77 percent of patients taking the combination achieved a reduction of more than 15 percent in ALP at 12 months, compared to 29 percent taking UDCA alone.
The drug has received a conditional marketing authorisation in Europe for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA.
According to the firm, its availability addresses an important unmet need as up to 40 percent of patients may have an inadequate response to UDCA, the only other available treatment for this condition.
"Despite the availability of UDCA, many patients have remained at significant risk of adverse outcomes with no alternative treatment option available. Ocaliva can now help fill an important unmet need for these patients," noted Frederik Nevens, University Hospitals Leuven & KU Leuven, Belgium, and the lead investigator of the Phase III POISE clinical study.
"As a community, our priority is to advocate for changes which ensure that people diagnosed with PBC have the best possible prognosis," said Tatjana Reic, president of the European Liver Patients Association (ELPA). "With this in mind, we are excited about this advance for patients with an inadequate response or intolerability to the current available treatment."
As per the terms of conditional clearance Intercept is required to provide post-approval updates on safety and efficacy analyses for Ocaliva from the ongoing Phase 4 COBALT outcomes study and a short-term study in patients with hepatic impairment.